Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Public ClinicalTrials.gov record NCT06567769. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Study identification
- NCT ID
- NCT06567769
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- GC Biopharma Corp
- Industry
- Enrollment
- 9 participants
Conditions and interventions
Conditions
Interventions
- GC1130A Drug
Drug
Eligibility (public fields only)
- Age range
- 12 Months to 18 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 20, 2024
- Primary completion
- May 31, 2027
- Completion
- May 31, 2027
- Last update posted
- Aug 27, 2025
2024 – 2027
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | Recruiting |
| University of Minnesota | Minneapolis | Minnesota | 55455 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06567769, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 27, 2025 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06567769 live on ClinicalTrials.gov.